FDA - NIH workshop on antisense oligonucleotide therapies

27 - 28 Sep 2010

Washington, DC, USA

An FDA and NIH co-sponsored meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases took place on the 27th and 28th September, 2010 in Washington DC, USA.

Given the growing number of AONs entering clinical development for multiple different neuromuscular disorders, the FDA and NIH, in collaboration with the research and advocacy communities, are taking a proactive role in developing and promoting regulatory science for the AONs by providing a forum for neuromuscular disease stakeholders to present the 'state-of-the-science' and exchange information on issues relevant to the AONs.

The meeting focused on four sessions:

  • Toxicology and Preclinical Findings to Date
  • Biomarkers
  • Clinical Trial Outcomes
  • Patient Registries and Assessing Long-Term Outcomes

and was concerned with four neuromuscular disorders:

  • Amyotrophic Lateral Sclerosis
  • Duchenne Muscular Dystrophy
  • Myotonic Dystrophy
  • Spinal Muscular Atrophy


The goal of this meeting was to allow stakeholders to explore potential pathways forward for the AONs with the eventual goal of creating a sound scientific anchoring for neuromuscular disease clinical development programs. These initial discussions should be seen as first steps in what is likely to be an iterative process intended to lay the ground work for future collaborations.

Over 120 stakeholders were invited to participate in the meeting, consisting of researchers, academics, industry and patient group representatives. In order to accommodate all those unable to attend the meeting due to space limitations or geographical distance, the whole meeting was also streamed on the web via a dedicated weblink.

A full meeting report is being produced and will be made available shortly. In the meantime, for any specific queries on the meeting please contact Abby Bronson or Emma Heslop.

To download the full agenda please click here.

The Steering Committee Chairs

Elizabeth McNeil, FDA
John Porter, NIH
Kate Bushby, TREAT-NMD
Ed Connor, CNMC